PhRMA supports the development of high quality information about patients’ medical treatment options. Empowering patients and physicians with high quality information will help ensure that our health system efficiently delivers the best possible results for all patients. Research from a range of sources in the public and private sectors provides valuable information for patients and health care professionals in making treatment decisions.
The following principles establish a framework to help ensure government-supported health outcomes research, including research on comparative effectiveness, meets patients’ needs and supports continued improvement in medical care. Research that meets these principles can improve health care decision-making, while research inconsistent with these principles in how it is designed or used will make it more difficult for patients to obtain the best and most efficient medical care.
I. Conduct research in the context of health care quality improvement: Government support of health outcomes research should occur as part of a broad agenda to improve health care quality and patient outcomes across the health care system. Efforts focused on cost-containment alone often shift cost from one medical service to another without improving patient outcomes. A research program geared toward improving quality and patient outcomes across the health care system is consistent with good research practice and answers the key question of how to most efficiently achieve better results for patients.
II. Prioritize to meet societal needs: Government priorities for health outcomes research should address diseases and conditions that are recognized as imposing a high clinical and economic burden on patients and society, rather than being limited to high cost interventions.
III. Consider the full range of treatment options: Health outcomes research should consider the full range of health care interventions and evaluate total health care costs or savings, not just costs of specific treatments.
IV. Support appropriate use of health care interventions: Failure to use interventions appropriately carries costs to patients and society. Research should encourage more appropriate use of health interventions, examining the widespread failure to use medical technologies appropriately as well as the overuse and misuse of technologies.
V. Consider the broad effects of health interventions: Health care interventions can have a wide range of costs and benefits. In evaluating outcomes of particular interventions, government-supported research should include direct benefits and broader and indirect benefits that are important to patients and society, such as quality of life, patient functionality and economic productivity.
VI. Keep research standards up to date: Health outcomes research is a growing and changing area of inquiry. Government-supported research in this area should stay at the forefront of knowledge in the field.
VII. Design and conduct quality research: Government-supported health outcomes research should use well-established standards for high-quality research, including making studies sufficiently powered (e.g., large enough patient groups) to provide sound results. Studies should report the strengths and limitations of the data and results, and conclusions should be consistent with these strengths and limitations.
VIII. Support physician and patient decision-making: Patients have unique medical needs that are not reflected by population-level research, requiring that patients and their health care professionals have the central role in making medical treatment decisions. Communication and use of research findings should explicitly recognize that health outcomes research is just one of many tools to help inform patients’ and doctors’ treatment choices.
IX. Recognize the benefits of multiple treatment options: Access to multiple therapies is needed to ensure that individual patients are optimally treated. Government-sponsored research also should acknowledge the potential economic benefits of having multiple treatment options, the ways that innovation can alter cost inputs such as prices over time, and the fact that medical progress often is achieved through a series of incremental improvements that cumulatively represent significant advances over time.
X. Support patient access to new health technologies: Health outcomes research findings should be used to help make more informed treatment decisions, not as a barrier that restricts or delays access to treatment choices. Health outcomes research is best applied to health care technologies after they have been used in a variety of clinical settings in the real world over an adequate period of time. Requiring health outcomes data before making a new technology available to patients would create significant delays in access and discourage innovation.
XI. Examine patient subgroups to meet individual patient needs and optimize value: Health outcomes research should consider and reflect the needs of patient subpopulations, who often respond differently to medicines based on factors such as age, genetic variation, and comorbidities. Because patient sub-populations can differ in their response to therapy, a variety of treatment options may be required to optimize treatment and provide the most clinical benefit and the greatest value.
XII. Seek public input from stakeholders: Government decisions related to the funding and conduct of health outcomes research should be made through open, transparent procedures that ensure appropriate and timely involvement of stakeholders such as patients, health care professionals and medical researchers on issues such as proposed research goals, study designs and communication of study results.
XIII. Facilitate communication of findings: The government should help ensure that consumers, patients, physicians and other health care professionals have access to the range of peer-reviewed results on all treatment options in a fair and balanced manner that is understandable to each group.